31 PROMs in autosomal inherited bleeding disorders: A systematic literature review 2 Patient-reported outcomes The included studies focused on several different PROs, such as HRQoL, physical functioning and pain. Table 2 provides an overview of PROs of included studies. Patient-reported outcomes in von Willebrand disease Health-related quality of life Three studies assessed HRQoL in adult patients with VWD 25,38,40 (Table 2). Compared to the general population, patients with VWD reported lower HRQoL, with the largest measured effects in the domains vitality, social functioning and pain 38,40. Barr et al. found that female patients scored lower compared to men in the domains emotion, cognition and pain 38. Two studies found a difference between the type of VWD and HRQoL measured 25,40. De Wee et al. found that patients with VWD type 3 scored significantly lower on the physical part of HRQoL and higher on pain levels compared to patients with VWD type 1 and 2 25. The study by Xu et al. supports this finding and also found that patients with VWD type 3 scored significantly lower on the physical domain of HRQoL compared to patients with VWD type 2 40. In contrast, two other studies found no difference in HRQoL between patients with VWD type 1, 2 and 3 38,39. One study 24 assessed HRQoL in children with VWD. They found that both preschool and school children had lower HRQoL compared to the general population in the domain general health perception. No difference was observed in HRQoL reported by parents of preschool children across the three types of VWD. In school children, parents of patients with VWD type 3 reported significantly lower HRQoL compared to the general population and patients with VWD type 1 and 2. No significant difference was found in HRQoL between boys and girls in both preschool and school children. 24 Self-perceived physical functioning Four studies 26-28,37 measured the influence of arthropathy and joint bleeds on self-perceived physical functioning in adult patients with VWD. Patients with a history of joint bleeds scored lower on functional abilities 26,28,37. The Von Willebrand in the Netherlands (WiN)-study found that patients with joint bleeds reported a lower overall HRQoL, especially with regard to social participation and physical limitation. In addition, they experienced more pain compared to patients without joint bleeds. 26,27
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